CA2940513C - Delivrance d'une proteine a domaine de recrutement des caspases (card) en tant que therapie pour inflammation oculaire - Google Patents

Delivrance d'une proteine a domaine de recrutement des caspases (card) en tant que therapie pour inflammation oculaire Download PDF

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Publication number
CA2940513C
CA2940513C CA2940513A CA2940513A CA2940513C CA 2940513 C CA2940513 C CA 2940513C CA 2940513 A CA2940513 A CA 2940513A CA 2940513 A CA2940513 A CA 2940513A CA 2940513 C CA2940513 C CA 2940513C
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Prior art keywords
protein
vector
tatcard
viral vector
use according
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CA2940513A
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CA2940513A1 (fr
Inventor
Cristhian J. ILDEFONSO
Alfred S. Lewin
Qiuhong Li
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University of Florida Research Foundation Inc
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University of Florida Research Foundation Inc
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • C07K14/4703—Inhibitors; Suppressors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0048—Eye, e.g. artificial tears
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/02—Fusion polypeptide containing a localisation/targetting motif containing a signal sequence
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/03—Fusion polypeptide containing a localisation/targetting motif containing a transmembrane segment
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/10—Fusion polypeptide containing a localisation/targetting motif containing a tag for extracellular membrane crossing, e.g. TAT or VP22
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Organic Chemistry (AREA)
  • Epidemiology (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Engineering & Computer Science (AREA)
  • Ophthalmology & Optometry (AREA)
  • Biotechnology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Zoology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Toxicology (AREA)
  • Rheumatology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Pain & Pain Management (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)

Abstract

La présente invention concerne des procédés et des compositions pour le traitement et/ou la prévention de la dégénérescence maculaire liée à l'âge et d'autres affections impliquant une dégénérescence, une néovascularisation oculaire ou une inflammation oculaire. Dans un exemple de mode de réalisation, l'invention concerne un procédé qui implique l'administration d'un vecteur d'expression qui délivre une CARD pouvant être sécrétée et pénétrant les cellules à un sujet nécessitant le traitement ou la prévention de la dégénérescence maculaire liée à l'âge et d'autres affections impliquant une dégénérescence ou une néovascularisation oculaire.
CA2940513A 2013-03-11 2014-03-11 Delivrance d'une proteine a domaine de recrutement des caspases (card) en tant que therapie pour inflammation oculaire Active CA2940513C (fr)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201361776076P 2013-03-11 2013-03-11
US61/776,076 2013-03-11
PCT/US2014/023262 WO2014164703A1 (fr) 2013-03-11 2014-03-11 Délivrance d'une protéine card en tant que thérapie pour inflammation oculaire

Publications (2)

Publication Number Publication Date
CA2940513A1 CA2940513A1 (fr) 2015-10-09
CA2940513C true CA2940513C (fr) 2023-08-15

Family

ID=51658947

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2940513A Active CA2940513C (fr) 2013-03-11 2014-03-11 Delivrance d'une proteine a domaine de recrutement des caspases (card) en tant que therapie pour inflammation oculaire

Country Status (5)

Country Link
US (3) US10981961B2 (fr)
EP (1) EP2968613B1 (fr)
AU (1) AU2014249045A1 (fr)
CA (1) CA2940513C (fr)
WO (1) WO2014164703A1 (fr)

Families Citing this family (10)

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WO2014140925A2 (fr) 2013-03-10 2014-09-18 Peritech Pharma Ltd. Compositions topiques et procédés de traitement de troubles topiques
WO2014164703A1 (fr) 2013-03-11 2014-10-09 University Of Florida Research Foundation, Inc. Délivrance d'une protéine card en tant que thérapie pour inflammation oculaire
US11053291B2 (en) 2014-02-19 2021-07-06 University Of Florida Research Foundation, Incorporated Delivery of Nrf2 as therapy for protection against reactive oxygen species
AU2015229381B2 (en) 2014-03-11 2019-11-07 University Of Florida Research Foundation, Inc. Use of AAV-expressed M013 protein as an anti-inflammatory therapeutic
WO2021050789A1 (fr) 2019-09-10 2021-03-18 Obsidian Therapeutics, Inc. Protéines de fusion de ca2-il15 pour une régulation accordable
WO2021216993A1 (fr) * 2020-04-24 2021-10-28 Pact Pharma, Inc. Méthodes de détermination d'efficacités d'édition de gènes dans des cellules
WO2021231538A2 (fr) * 2020-05-13 2021-11-18 Akouos, Inc. Compositions et méthodes de traitement de perte auditive associée à kcnq4
US20240197895A1 (en) * 2020-12-22 2024-06-20 The Trustees Of Columbia University In The City Of New York Compositions and methods for treating patients with mitochondrial complex i deficiency using caspase-9 signaling pathway inhibitors
EP4581044A1 (fr) 2022-08-31 2025-07-09 Unm Rainforest Innovations Compositions ciblant une protéine de type speck associée à l'apoptose présentant un domaine d'activation et de recrutement de caspase (asc) et procédés d'utilisation
WO2025081130A1 (fr) * 2023-10-12 2025-04-17 University Of Miami Utilisation d'anticorps anti-asc dans le traitement de troubles oculaires

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US11739130B2 (en) 2023-08-29
EP2968613A1 (fr) 2016-01-20
US20240254178A1 (en) 2024-08-01
HK1220613A1 (en) 2017-05-12
AU2014249045A1 (en) 2015-10-29
US12415841B2 (en) 2025-09-16
WO2014164703A1 (fr) 2014-10-09
US20210371481A1 (en) 2021-12-02
EP2968613B1 (fr) 2019-09-11
US20160017012A1 (en) 2016-01-21
US10981961B2 (en) 2021-04-20
EP2968613A4 (fr) 2016-10-19
US20230132355A2 (en) 2023-04-27

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